- Gene therapy is being investigated as a possible treatment for individuals with immunodeficiency diseases or other conditions that make it difficult to fight off infection. Gene therapy avoids problems with donor identification and possible rejection of bone marrow transplant by using the patient s own modified blood cells to help treat the disease. Researchers are interested in collecting stem cells from the blood of individuals with immunodeficiency diseases in order to use the cells to develop potential gene therapy treatments.

Objectives:

- To collect blood stem cells from patients with immunodeficiency diseases tto test our ability to correct the defects of these cells in the test tube. .

Eligibility:

- Individuals between 18 and 40 years of age with immunodeficiency diseases.

- Individuals with human immunodeficiency virus (HIV) will not be able to participate in this study.

Design:

- Participants will provide an initial blood sample for disease screening (such as hepatitis B and C, syphilis, or viruses like the Epstein-Barr virus, herpes simplex virus, or toxoplasmosis) and to check kidney and liver function.

- Starting 5 days before blood donation, participants will receive daily injections of a drug called G-CSF (granulocyte colony stimulating factor, or filgrastim), which pushes stem cells out of the bone marrow and into the bloodstream. Participants will receive the injections at the National Institutes of Health Clinical Center.

- On day 5, participants will have a single leukapheresis procedure to collect the stem cells from the blood.

- No additional treatment will be provided as part of this protocol. The cells that are collected will be used fore experiments in the lab and will not be used to treat individuals with these diseases.

f) Adequate venous access for peripheral apheresis, or consent to use a temporary central venous catheter for apheresis.

g) Female patients of childbearing age must have a negative urine pregnancy test within one week of beginning G-CSF administration.

h) A patient who is lactating must be willing and able to interrupt breast-feeding or substitute formula feeding for her infant during the period of filgrastim administration and for two days following the final dose. Filgrastim may be secreted in human milk, although its bioavailability from this source is not known. Limited clinical data suggest that short-term administration of filgrastim or sargramostim to neonates is not associated with adverse outcomes

j) Potential patients must be screened by an apheresis nurse to check venous access before protocol entry. Antecubital veins must be adequate for peripheral access during leukapheresis, or the patient must give written consent for placement of a temporary central venous access catheter.

EXCLUSION CRITERIA:

a) HIV infection.

b) Chronic hepatitis B or hepatitis C virus infection.

c) History of psychiatric disorder which may compromise compliance with protocol, or which does not allow for appropriate informed consent.

d) Active infection that is not responding to antimicrobial therapy.

f) Pregnant. The effects on breast-milk are also unknown and may be harmful to the infant; therefore, women should not breast feed during the interval from study entry to collection.

g) Sexually active individuals capable of becoming pregnant who are unable or unwilling to use effective form(s) of contraception during time enrolled on study. Effective forms of contraception include one or more of the following: intrauterine device (IUD), hormonal (birth control pills, injections, or implants), tubal ligation/hysterectomy, partner s vasectomy, barrier methods, (condom, diaphragm, or cervical cap), or abstinence. Males on the protocol must use an effective form of contraception at study entry.

h) Presence of active malignancy in another organ system other than the hematopoietic system.

i) History of hypertension that is not controlled by medication, stroke, or severe heart disease. Individuals with symptomatic angina will be considered to have severe heart disease and will not be eligible.